P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation
Year: 2024
Type: article
Study design: Study Protocol
Abstract: Mucopolysaccharidosis type I Hurler (MPS-IH) is an autosomal recessive lysosomal storage disorder characterized by deficiency of alpha-L-iduronidase (IDUA), which is responsible for the degradation of glycosaminoglycans (GAGs). The accumulation of GAGs throughout the body, including the brain, results in tissue damage and multi-organ dysfunction causing a wide range of musculoskeletal, cardiopulmonary, ophthalmic and auditory abnormalities, progressive neurologic disease, and early death. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the current standard of care (SoC) but is associated with significant complications and residual disease burden, having a limited effect on skeletal, ophthalmic, cardiac, and other manifestations of MPS-IH.
Source: Genetics in Medicine Open
Institutions: University of California, San Francisco, Manchester University NHS Foundation Trust, Royal Manchester Children's Hospital, University of Minnesota, Utrecht University, Princess Máxima Center, Children's Hospital of Philadelphia, University Medical Center Utrecht, Wilhelmina Children's Hospital, The San Raffaele Telethon Institute for Gene Therapy, Orchard Therapeutics (United Kingdom)
Language: English
Subfield: Physiology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $3,200
OpenAlex ID: W4392616491